Fig. 2.

Enhanced IDUA expression by IDUA-HSPCs and derived macrophages. a Representative FACS plot showing three distinct populations based on YFP expression 3 days post-modification. b Percent YFP-positive cells in culture (30 days). c, Fold increase in IDUA secretion and intracellular expression by YFP-high, YFP-low, and YFP-negative populations compared to mock cells. d, Average LAMP-1 + area in MPSI fibroblasts co-cultured with IDUA-HSPCs. Each dot represents a cell. e, Human CD34, CD14, and CD11b marker expression in HSPC-derived macrophages after in vitro differentiation compared to undifferentiated cells (CD34 + HSPCs). Macrophage morphology and YFP expression after differentiation. f, Fold increase in IDUA secretion and intracellular expression in HSPC-macrophages modified with SFFV and PGK expression cassettes. c, e, f, Each column represents average of triplicates in n = 3 independent biological samples. All data expressed as mean ± SD, ***p < .001 in two-sided unpaired t-test. Source data are provided as a Source Data file